A - Human Necessities – 61 – K
Patent
A - Human Necessities
61
K
A61K 31/00 (2006.01) A61K 31/137 (2006.01) A61P 43/00 (2006.01)
Patent
CA 2699864
Therapeutic compositions and methods for treatment of late-onset Gaucher disease are described herein. The compositions comprise compounds having activity as pharmacological chaperones for mutant forms of the beta-glucocerebrosidase. Methods of treatment involve providing therapeutically effective amounts of such compositions to subjects in need thereof.
L'invention porte sur des compositions thérapeutiques et sur des procédés pour le traitement de la maladie de Gaucher à début tardif. Les compositions incluent des composés présentant une activité en tant que chaperons pharmacologiques pour des formes mutantes de la bêta-glucocérébrosidase. L'invention porte sur des procédés de traitement qui mettent en jeu la fourniture de quantités thérapeutiquement efficaces de telles compositions à des sujets en ayant besoin.
Blanchard Jan E.
Brown Eric D.
Buttner Justin D.
Mahuran Don J.
Tropak Michael B.
Mccarthy Tetrault Llp
Mcmaster University
The Hospital For Sick Children
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